Article
Allele-specific silencing by RNAi of R92Q and R173W mutations in cardiac troponin T.
Experimental biology and medicine (Maywood, N.J.) - 1 May 2022
Migliore Loredana, Galvagni Federico, Pierantozzi Enrico, Sorrentino Vincenzo, Rossi Daniela
Abstract excerpt
Autosomal dominant mutations in sarcomere proteins such as the cardiac troponin T (TNNT2) are the main genetic causes of human hypertrophic cardiomyopathy and dilated cardiomyopathy. Allele-specific silencing by RNA interference (ASP-RNAi) holds promise as a therapeutic strategy for downregulating a single mutant allele with minimal suppression of the corresponding wild-type allele. Here, we propose ASP-RNAi as a...
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